Real-World Outcomes of Ruxolitinib Treatment for Polycythemia Vera.

Autor: Coltoff A; Department of Hematology/Oncology, Columbia New York Presbyterian, New York, NY., Mesa R; Mays Cancer Center, UT Health San Antonio MD Anderson, San Antonio, TX., Gotlib J; Department of Medicine, Division of Hematology, Cancer Institute, Stanford University, Stanford, CA., Shulman J; Department of Leukemia, Memorial Sloan Kettering Cancer Center, New York, NY., Rampal RK; Department of Leukemia, Memorial Sloan Kettering Cancer Center, New York, NY., Siwoski O; Division of Hematologic Malignancies and Cellular Therapeutics (HMCT), Department of Internal Medicine, University of Kansas Cancer Center, Westwood, KS., Yacoub A; Division of Hematologic Malignancies and Cellular Therapeutics (HMCT), Department of Internal Medicine, University of Kansas Cancer Center, Westwood, KS., Moliterno A; Division of Hematology, Department of Medicine, Johns Hopkins University School of Medicine, Baltimore, MD., Yang A; Division of Hematology, Department of Medicine, Johns Hopkins University School of Medicine, Baltimore, MD., Braunstein E; Division of Hematology, Department of Medicine, Johns Hopkins University School of Medicine, Baltimore, MD., Gerds AT; Leukemia and Myeloid Disorders Program, Cleveland Clinic, Cleveland, OH., Hobbs GS; Cancer Center, Massachusetts General Hospital Harvard Medical School, Boston, MA., Winton EF; Winship Cancer Institute, Emory University, Atlanta, GA., Goel S; Division of Hematology, Department of Oncology, Montefiore Medical Center, Albert Einstein College of Medicine, Bronx, NY., Wadleigh M; Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA., Tremblay D; Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY., Moshier E; Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY., Mascarenhas J; Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY. Electronic address: john.mascarenhas@mssm.edu.
Jazyk: angličtina
Zdroj: Clinical lymphoma, myeloma & leukemia [Clin Lymphoma Myeloma Leuk] 2020 Oct; Vol. 20 (10), pp. 697-703.e1. Date of Electronic Publication: 2020 May 29.
DOI: 10.1016/j.clml.2020.05.019
Abstrakt: Introduction: Ruxolitinib is approved for the treatment of polycythemia vera (PV) with hydroxyurea resistance or intolerance. Approval was based on the phase III RESPONSE trial, which demonstrated efficacy in a highly selected patient population.
Materials and Methods: To characterize the tolerability and outcomes of ruxolitinib outside of a clinical trial, we performed a multi-center retrospective analysis of patients with PV treated with ruxolitinib at 11 participating sites across the United States. Outcomes of interest included change in phlebotomy requirements after starting ruxolitinib and spleen response, as these were included in the primary composite outcome in the RESPONSE trial.
Results: One hundred twenty-six patients met eligibility criteria, and the median duration of follow-up was 22.4 months (range, 0-63.0 months). At 32 weeks after starting ruxolitinib, the percentage of patients who received at least 1 phlebotomy was significantly decreased compared with before ruxolitinib (37% vs. 56%; relative risk [RR], 0.66; 95% confidence interval [CI], 0.52-0.84; P < .001). Phlebotomy requirements were similarly decreased in patients who had received at least 3 phlebotomies prior to ruxolitinib initiation (28% vs. 17%; RR, 1.65; 95% CI, 1.13-2.40; P < .01). Resolution of palpable splenomegaly was also documented (48% vs. 20%; RR, 2.45; 95% CI, 1.70-3.53; P < .0001). A total of 9.5% of patients discontinued ruxolitinib owing to treatment-emergent adverse events, and 81.7% of patients were receiving ruxolitinib at last known follow-up.
Conclusion: These real-world results are similar to those reported from the RESPONSE trial, although additional follow-up is necessary to assess long-term outcomes and potential for late-onset toxicity.
(Copyright © 2020 Elsevier Inc. All rights reserved.)
Databáze: MEDLINE
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